KR20240063110A - 치료제의 역행성 관상 정맥 또는 정맥동 투여 - Google Patents

치료제의 역행성 관상 정맥 또는 정맥동 투여 Download PDF

Info

Publication number
KR20240063110A
KR20240063110A KR1020247007654A KR20247007654A KR20240063110A KR 20240063110 A KR20240063110 A KR 20240063110A KR 1020247007654 A KR1020247007654 A KR 1020247007654A KR 20247007654 A KR20247007654 A KR 20247007654A KR 20240063110 A KR20240063110 A KR 20240063110A
Authority
KR
South Korea
Prior art keywords
therapeutic agent
vector
administered
heart
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020247007654A
Other languages
English (en)
Korean (ko)
Inventor
아서 엠. 펠드먼
발레리 마이어스
마르시아 볼로냐
Original Assignee
템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션
리노바코, 인코포레이티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션, 리노바코, 인코포레이티드 filed Critical 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션
Publication of KR20240063110A publication Critical patent/KR20240063110A/ko
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/12Antihypertensives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/02Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hospice & Palliative Care (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020247007654A 2021-08-12 2022-08-12 치료제의 역행성 관상 정맥 또는 정맥동 투여 Pending KR20240063110A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163260225P 2021-08-12 2021-08-12
US63/260,225 2021-08-12
US202163263442P 2021-11-02 2021-11-02
US63/263,442 2021-11-02
US202263343985P 2022-05-19 2022-05-19
US63/343,985 2022-05-19
PCT/US2022/074944 WO2023019272A1 (en) 2021-08-12 2022-08-12 Retrograde coronary venous or sinus administration of therapeutics

Publications (1)

Publication Number Publication Date
KR20240063110A true KR20240063110A (ko) 2024-05-10

Family

ID=85201030

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247007654A Pending KR20240063110A (ko) 2021-08-12 2022-08-12 치료제의 역행성 관상 정맥 또는 정맥동 투여

Country Status (10)

Country Link
US (1) US20260069719A1 (https=)
EP (1) EP4384269A4 (https=)
JP (1) JP2024529170A (https=)
KR (1) KR20240063110A (https=)
AU (1) AU2022325237A1 (https=)
CA (1) CA3228581A1 (https=)
CL (1) CL2024000397A1 (https=)
IL (1) IL310763A (https=)
MX (1) MX2024001939A (https=)
WO (1) WO2023019272A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015117010A2 (en) 2014-01-31 2015-08-06 Temple University Of The Commonwealth System Of Higher Education Bag3 as a target for therapy of heart failure
WO2024229259A1 (en) * 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015117010A2 (en) * 2014-01-31 2015-08-06 Temple University Of The Commonwealth System Of Higher Education Bag3 as a target for therapy of heart failure
SG11201803934YA (en) * 2015-11-11 2018-06-28 Intrexon Corp Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies

Also Published As

Publication number Publication date
AU2022325237A1 (en) 2024-03-21
IL310763A (en) 2024-04-01
AU2022325237A2 (en) 2024-03-28
EP4384269A4 (en) 2026-03-18
CL2024000397A1 (es) 2024-08-30
JP2024529170A (ja) 2024-08-01
EP4384269A1 (en) 2024-06-19
US20260069719A1 (en) 2026-03-12
CA3228581A1 (en) 2023-02-16
MX2024001939A (es) 2024-07-10
WO2023019272A1 (en) 2023-02-16

Similar Documents

Publication Publication Date Title
US20250066813A1 (en) Means and method for preparing viral vectors and uses of same
JP7607007B2 (ja) 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
RU2653444C2 (ru) Композиции вектора aav и способы переноса генов в клетки, органы и ткани
KR20210148273A (ko) 근육 발현을 위한 하이브리드 프로모터
CN114466682A (zh) 用于治疗基于肌营养不良蛋白的肌病的靶向外显子44的核酸和包含所述核酸的重组腺相关病毒
US20260069719A1 (en) Retrograde coronary venous or sinus administration of therapeutics
US20230175015A1 (en) Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
US12415850B2 (en) Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
CN118591396A (zh) 治疗剂的逆行冠状静脉或冠状窦施用
RU2818529C2 (ru) Средства и способ получения вирусных векторов и варианты их применения
WO2024259064A1 (en) Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
KR20260052185A (ko) 바이러스 벡터를 제조하기 위한 수단 및 방법 및 그의 용도
HK1223849B (en) Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000